Casgevy FDA Approval History
Last updated by Judith Stewart, BPharm on Jan 17, 2024.
FDA Approved: Yes (First approved December 8, 2023)
Brand name: Casgevy
Generic name: exagamglogene autotemcel
Dosage form: Suspension for Intravenous Infusion
Company: Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics
Treatment for: Beta Thalassemia, Sickle Cell Disease
Casgevy (exagamglogene autotemcel) is a CRISPR/Cas9 genome-edited cell therapy for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia.
- Casgevy is indicated for the treatment of patients aged 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs), and transfusion-dependent beta-thalassemia (TDT).
- Sickle cell disease is a common genetic disease caused by mutations in the HBB gene encoding the beta chain of adult hemoglobin (HbA). It is characterized by red blood cells that become hard and sticky and look like a C-shaped "sickle," causing pain, anemia and other complications.
- Beta-thalassemia is a genetic disease also caused by mutations in the HBB gene. Transfusion-dependent beta-thalassemia is the most severe type of beta-thalassemia and TDT patients usually require frequent blood transfusions and iron chelation therapy.
- Casgevy contains exagamglogene autotemcel which is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth.
- Casgevy has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.
- Treatment with Casgevy involves a patient having their own hematopoietic stem and progenitor cells collected from peripheral blood, which are then edited using CRISPR/Cas9 technology. The edited cells are then infused back into the patient as part of an autologous hematopoietic stem cell transplant (HSCT) after myeloablative busulfan conditioning.
- Warnings and precautions associated with Casgevy include potential neutrophil engraftment failure, prolonged time to platelet engraftment, hypersensitivity reactions, and off-target genome editing risk.
- Common adverse reactions in SCD patients include mucositis, febrile neutropenia, and decreased appetite.
Common adverse reactions in TDT patients include mucositis and febrile neutropenia.
Common Grade 3 or 4 laboratory abnormalities include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia. - Casgevy was approved for use in the treatment of sickle cell disease on December 8, 2023 and became the first medicine available in the United States to treat a genetic disease using the CRISPR gene-editing technique. Casgevy was approved for use in the treatment of transfusion-dependent beta thalassemia on January 16, 2024.
Development timeline for Casgevy
Further information
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